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AAV CRISPR - Crossref

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Last Updated: 23 April 2022

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Inherent hepatocytic heterogeneity determines expression and retention of edited F9 alleles post-AAV/CRISPR infusion

In determining the therapeutic value of this therapy, a key role of host cell heterogeneity was determined by systemic analysis, wherein a suboptimal inflammation response regulated the P2 activity and the retention of the edited F9 alleles in a hepatocytic sub-dependent manner. These results collectively highlight the key factors that influence the success and longevity of AAV/CRIPSR-mediated gene editing in dealing with complex hepatic monogenic disorders.

Source link: https://doi.org/10.1073/pnas.2110887118


AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa

Retinitis pigmentosa is a group of chronic, retinal degenerative disorders that can result in vision loss or blindness in advanced stages. Gene therapy has been particularly effective in treating autosomal recessive RP. In vivo gene editing may be a therapeutic option to treat adRP.

Source link: https://doi.org/10.3390/pharmaceutics14040824


CRISPR Systems Suitable for Single AAV Vector Delivery

A small Cas orthologs that can be packaged, along with the required guide RNA elements, will be a useful improvement for CRISPR/-Cs gene editing. This review examines the benefits and current status of small Cas protein orthologs that are suitable for gene editing approaches using single AAV vector delivery.

Source link: https://doi.org/10.2174/1566523221666211006120355


Abstract 032: Somatic Editing of Ldlr with AAV-CRISPR for Atherosclerosis Studies

Background: Current research into genetic causes of atherosclerosis are both time-consuming and expensive. Hypothesis: A live-directed somatic disruption of Ldlr using an all-in-one AAV-CRISPR vector can produce atherosclerotic lesions in adult mice, eliminating the need to cross to Ldlr KO mice. Methods: An Adeno-Associated Viral vector based on serotype 8 was used to provide Staphylococcus Cas9 and a small guide RNA targeting the Ldlr gene. Adult C57BL6/J mice were sent either: 1 saline, 2 AAV-CRISPR, or 3 AAV-hPCSK9, versus 4 germline Ldlr KO mice. With AAV-CRISPR and AAV-hPCSK9 relative to germline Ldlr KO mice, the incidence of lesion burden was marginally smaller. In summary, our all-in-one AAV-CRISPR vector is a cost-effective alternative to the use of Ldlr KO mice to study atherosclerosis.

Source link: https://doi.org/10.1161/atvb.38.suppl_1.032


In Vivo AAV-CRISPR/Cas9–Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

Methods: The aim of this study was to determine whether in vivo somatic cell gene editing by adeno-associated virus in a mouse model could treat familial hypercholesterolemia caused by the Ldlr mutant's familial hypercholesterolemia caused by the Ldlr mutant. Conclusions: We found that homogeneous Ldlr E208X mice developed severe atherosclerotic phenotypes after a high-fat diet, and that the Ldlr mutation was corrected in a subset of hepatocytes following AAV-CRISPR/Cas9 treatment, with LDLR protein expression partially restored. Conclusions: Our study indicates that in vivo AAV-CRISPR/Cas9-mediated Ldlr gene repair can partially restore LDLR expression and inherently improve atherosclerosis phenotypes in Ldlr mutants, providing a potential therapeutic strategy for patients with familial hypercholesterolemia.

Source link: https://doi.org/10.1161/circulationaha.119.042476


Antiproliferative effects of AAV-delivered CRISPR/Cas9-based degradation of the HPV18-E6 gene in HeLa cells

Human papillomavirus infections are responsible for the majority of cervical cancers, which are the fourth most common cancer in women. We found that disruption of the HPV-E6 gene resulted in increased cell apoptosis and reduced cell proliferation in our results. In the cell profiling assay, there was a significant accumulation of infected cells in sub-G1 phase. Our results showed that AAV-mediated delivery of CRISPR/Cas9 can safely target the HPV-E6 gene in HeLa cells, as well as its anti-inflammatory properties, and local administration of this gene-editing device for HPV-related cervical cancers can be helpful in treating local administration of this gene-editing device.

Source link: https://doi.org/10.1038/s41598-022-06025-w


Co-editing PINK1 and DJ-1 genes via AAV-delivered CRISPR/Cas9 system in adult monkey brains elicits classic Parkinsonian phenotypes

Abstract Whether direct manipulation of Parkinson's disease risk genes in monkey brain could produce Parkinson's disease risk genes remains a unsolved problem. This gene editing device can be used to produce a large number of genetically edited PD monkeys in a short time, providing a realistic transgenic monkey model for future PD research.

Source link: https://doi.org/10.1101/2020.09.19.305003

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions

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* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions